What You Need to Know About Homology Medicines

Homology Medicines was founded and incubated with a seed investment within the 4:59 Initiative, the company creation engine of 5AM Ventures. Homology is building on the naturally occurring process of homologous recombination.

The company has an exclusive worldwide license to its technology platform, which is based on the research of Saswati Chatterjee, Department of Surgery, member of the Beckman Research Institute at the City of Hope in California. The company’s co-founders are Saswati Chatterjee and Laura Smith, a postdoctoral fellow in the AAV Lab and the Department of Surgery.

“Homology’s ability to attract top management, who over many years have gained scientific and practical knowledge of the complexities of developing treatment for rare genetic diseases, is a testament to the strength of our technology platform,” said Kush Parmar, 5AM Ventures’ managing partner, in a May statement. “The company has an aggressive development plan and a significant opportunity to leapfrog current strategies in gene therapy and editing as it builds an expansive and high-value clinical pipeline.”

The company believes it has a type of gene editing technology that is safer and more effective than other methods, most specifically CRISPR-Cas9.

Company LeadershipArthur Tzianabos—president and chief executive officer. Prior to joining Homology, Tzianabos was president and chief scientific officer for OvaScience . He spent nine years at Shire Pharmaceuticals .

Sam Rasty—chief operating officer. Most recently, Rasty was vice president and head of new products at the Rare Diseases business unit of Shire Pharmaceuticals.

Albert Seymour—chief scientific officer. Prior to joining Homology, Seymour was senior vice president and Global Head of Research and Nonclinical Development at Shire .

Company Financing
Homology launched in May 2016 with a $43.5 million Series A preferred stock financing. It was co-led by 5AM Ventures and ARCH Venture Partners. Additional investors included Temasek, Deerfield Management, and ARCH Overage Fund.

Pipeline
Chatterjee and her team led the first adeno-associated virus (AAV) vector-mediated gene transfer studies into human hematopoietic stem cells. They subsequently identified and isolated a series of naturally-occurring AAVs from human CD34+ cells.

The company’s approach is called AMEnDR (AAV-Mediated Editing by Directed Homologous Recombination), which can correct and edit a gene mutation through in vivo delivery of a single AAV vector.

Dollars and Deals
On September 15, Homology licensed worldwide rights to a novel in vivo AAV technology from the California Institute of Technology. It plans to combine Caltech’s AAV technology with its own next-generation in vivo gene editing and gene transfer technology to develop treatments for central nervous system (CNS) disorders.

Under the deal, Homology has a co-exclusive license to specific patented AAV capsid sequences and peptides that have shown the ability to cross the blood brain barrier. The company also has a non-exclusive license to the patented methodology for in vivo screening, engineering and enrichment of novel AAV capsids.

What to Look For
The company plans to start with rare genetic diseases, possible sickle cell disease.